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Nucleic Acid (9 companies)

Ticker Company Therapeutic areas Programs Lead stage Programs
AMLX Amylyx Pharmaceuticals, Inc. MetabolicNeurology Rare 1 Phase 3 5
BEAM Beam Therapeutics Inc. HematologyMetabolicRespiratory Rare 3 Phase 1 8
DYN Dyne Therapeutics, Inc. MetabolicNeurology Rare 9 Phase 3 10
PHIO Phio Pharmaceuticals Corp. Oncology 4 Phase 1 4
SRPT Sarepta Therapeutics, Inc. Neurology Rare 5 Approved 10
RARE Ultragenyx Pharmaceutical Inc. CardiovascularGI/HepatologyMetabolicNeurology Rare 1 Approved 12
VNDA Vanda Pharmaceuticals Inc. CardiovascularDermatologyGI/HepatologyHematologyImmunologyInfectious DiseaseNeurologyOphthalmology Rare 2 Approved 26
VIR Vir Biotechnology, Inc. Infectious DiseaseOncology Rare 1 Phase 3 10
WVE Wave Life Sciences Ltd. MetabolicNeurologyRespiratory Rare 5 Phase 2 5